Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02267863
Recruitment Status : Recruiting
First Posted : October 20, 2014
Last Update Posted : March 25, 2021
Sponsor:
Information provided by (Responsible Party):
Aptose Biosciences Inc.

Brief Summary:
This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.

Condition or disease Intervention/treatment Phase
Acute Myelogenous Leukemia in Relapse Acute Myelogenous Leukemia, Relapsed, Adult Acute Myelogenous Leukemia, Adult Acute Myelogenous Leukemia High Risk Myelodysplasia Drug: APTO-253 Phase 1

Detailed Description:
This is a multicenter, open-label, Phase Ia/b dose escalation study of safety, pharmacodynamics, and pharmacokinetics of APTO-253 in ascending cohorts (3+3 design) to determine the MTD or recommended dose in patients with relapsed or refractory acute myelogenous leukemia (AML) or high-risk MDS patients. This is to be followed by a cohort expansion phase at the MTD or recommended dose.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase Ia/b Dose Escalation and Expansion, Multicenter, Open-label, Safety, Pharmacokinetic and Pharmacodynamic Study of APTO-253 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia or High-Risk Myelodysplasia
Study Start Date : October 2014
Estimated Primary Completion Date : February 2022
Estimated Study Completion Date : May 2022


Arm Intervention/treatment
Experimental: Dose Escalation and Expansion
APTO-253 will be given in ascending doses in patients with relapsed or refractory AML or high risk MDS (escalation cohort), until the maximum tolerated dose or recommended dose is reached. Followed by up to 30 patients enrolled in the expansion cohort at the recommended dose.
Drug: APTO-253
APTO-253 will be given in ascending doses starting at 20 mg/m2 until the maximum tolerated dose or recommended dose is reached.




Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events of APTO-253 [ Time Frame: Cycle 1 (28 days) ]
    To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.

  2. Maximum tolerated dose and dose limiting toxicities [ Time Frame: Cycle 1 (28 days) ]
    To determine the maximum tolerated dose (MTD) and the dose limiting toxicities (DLT) of APTO-253 when given on days 1, 8, 15, and 22 of each 28-day cycle.

  3. Establish recommended dose for future development of APTO-253 [ Time Frame: Up to 7 months ]
    To establish the dose of APTO-253 recommended for future development of APTO-253 for patients with specific types of hematologic malignancies.


Secondary Outcome Measures :
  1. Pharmacokinetic variables including maximum plasma concentration (Cmax) [ Time Frame: Cycle 1 (28 days) ]
    Pharmacokinetic variables including maximum plasma concentration (Cmax)

  2. Pharmacokinetic variables including minimum plasma concentration (Cmin) [ Time Frame: Cycle 1 (28 days) ]
    Pharmacokinetic variables including minimum plasma concentration (Cmin)

  3. Pharmacokinetic variables including Area Under the Curve (AUC) [ Time Frame: Cycle 1 (28 days) ]
    Pharmacokinetic variables including Area Under the Curve (AUC)

  4. Pharmacokinetic variables including volume of distribution [ Time Frame: Cycle 1 (28 days) ]
    Pharmacokinetic variables including volume of distribution

  5. Pharmacokinetic variables including clearance [ Time Frame: Cycle 1 (28 days) ]
    Pharmacokinetic variables including clearance

  6. Pharmacokinetic variables including serum half-life [ Time Frame: Cycle 1 (28 days) ]
    Pharmacokinetic variables including serum half-life

  7. Assess for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS. [ Time Frame: Average 2 Cycles (8 weeks) ]
    To observe patients for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.

  8. Determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect. [ Time Frame: Average 2 Cycles (8 weeks) ]
    To determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients ≥18 years old
  • Life expectancy of at least 2 months
  • Off previous cancer therapy for at least 14 days, or 5 half-lives for noncytotoxic agents prior to first study treatment administration
  • Patients must have a calculated creatinine clearance >60 mL/min
  • Acceptable hematologic, renal and liver functions and coagulation status parameters

Exclusion Criteria:

  • Patients with GVHD requiring systemic immunosuppressive therapy
  • Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and clinical significant disease related metabolic disorder
  • Clinically significant intravascular coagulation
  • Treatment with other investigational drugs within 14 days prior to first study treatment administration

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02267863


Contacts
Layout table for location contacts
Contact: Ashleigh Campillo 858-333-6118 acampillo@aptose.com
Contact: Victor Montalvo Lugo 858-926-2740 VMontalvo@aptose.com

Locations
Layout table for location information
United States, Arizona
University of Arizona Cancer Center Terminated
Tucson, Arizona, United States, 85724
United States, California
UC San Diego Moores Cancer Center Recruiting
La Jolla, California, United States, 92093-0698
Contact: Jesika Reiner, MPH    858-822-5364    jreiner@ucsd.edu   
Principal Investigator: Dimitrios Tzachanis, MD, PhD         
University of California, Irvine Recruiting
Orange, California, United States, 92868
Contact    877-827-8839    ucstudy@uci.edu   
Contact: Blake Johnson    714-456-3476    blakej@uci.edu   
Principal Investigator: Deepa Jeyakumar, MD         
United States, Georgia
Emory University; Winship Cancer Institute Recruiting
Atlanta, Georgia, United States, 30322
Contact: Shannon Gleason, MLS    404-778-4334    shannon.gleason@emory.edu   
Contact: Victor Neirinckx    404-778-4994    victor.d.neirinckx@emory.edu   
Principal Investigator: Martha Arellano, MD         
United States, Louisiana
Ochsner Cancer Institute Withdrawn
New Orleans, Louisiana, United States, 70121
United States, Michigan
University of Michigan Completed
Ann Arbor, Michigan, United States, 48109
United States, Montana
St. Vincent Frontier Cancer Center Recruiting
Billings, Montana, United States, 59102
Contact: Heather Duyck    406-238-6290    heather.duyck@sclhealth.org   
Principal Investigator: Patrick Cobb, MD         
United States, New York
University of Rochester; Wilmot Cancer Institute Clinical Trials Office Recruiting
Rochester, New York, United States, 14643
Contact: Haley Misch    585-275-5345    Haley_Misch@urmc.rochester.edu   
Principal Investigator: Kristen ODwyer, MD         
United States, Ohio
University Hospital Withdrawn
Cleveland, Ohio, United States, 44106
United States, Oregon
Oregon Health & Science University Completed
Portland, Oregon, United States, 97239
United States, South Carolina
Prisma Health, Institute for Translational Oncology Research Recruiting
Greenville, South Carolina, United States, 29605
Contact: Jill Roemmich    864-455-3294    jill.roemmich@prismahealth.org   
Principal Investigator: Elizabeth Cull, MD         
United States, Texas
Baylor Research Institute Recruiting
Dallas, Texas, United States, 75246
Contact: Lisa Jones    214-820-1970    LISA.JONES2@BSWHEALTH.ORG   
Principal Investigator: Moshe Y. Levy, MD         
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Robin Thomas, RN, BSN    713-745-4642    RMThomas3@mdanderson.org   
Contact: Mary Kelly, RN    713-794-4656    MAKelly1@mdanderson.org   
Principal Investigator: Maro Ohanian, MD         
Sponsors and Collaborators
Aptose Biosciences Inc.
Investigators
Layout table for investigator information
Study Director: Rafael Bejar, MD., PhD. Aptose Biosciences Inc.
Additional Information:
Layout table for additonal information
Responsible Party: Aptose Biosciences Inc.
ClinicalTrials.gov Identifier: NCT02267863    
Other Study ID Numbers: 253-HEM1-01
First Posted: October 20, 2014    Key Record Dates
Last Update Posted: March 25, 2021
Last Verified: December 2020
Keywords provided by Aptose Biosciences Inc.:
AML
MDS
Leukemia
Acute Myeloid Leukemia
Aptose
APTO-253
Kinase Inhibitor
Additional relevant MeSH terms:
Layout table for MeSH terms
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Preleukemia
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions